VISION-DMD - Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy | VISION DMD | Project | Results | H2020 | CORDIS

Resultado final

Develop and publish project website including public summaries of activity

Completion of workshop series

Publicaciones

Pubertal induction in adolescents with DMD is associated with high satisfaction, gonadotropin release and increased muscle contractile surface area

Autores: Claire L Wood Kieren G Hollingsworth, Eric Hughes, Sadhanandham Punniyakodi, Robert Muni-Lofra, Anna Mayhew, Rod T Mitchell, Michela Guglieri, Timothy D Cheetham, and Volker Straub
Publicado en: European Journal of Endocrinology, Edición 2021;184(1), 2021, Página(s) 67-79., ISSN 1479-683X
Editor: European Society of Endocrinology
DOI: 10.1530/eje-20-0709

Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes

Autores: Eric P. Hoffman, Valerie Riddle, Maxime A. Siegler, Daniel Dickerson, Miroslav Backonja, William G. Kramer, Kanneboyina Nagaraju, Heather Gordish-Dressman, Jesse M. Damsker, John M. McCall
Publicado en: Steroids, 2018, ISSN 0039-128X
Editor: Elsevier BV
DOI: 10.1016/j.steroids.2018.02.010

Quantifying fat replacement of muscle by quantitative MRI in muscular dystrophy

Autores: Jedrzej Burakiewicz, Christopher D. J. Sinclair, Dirk Fischer, Glenn A. Walter, Hermien E. Kan, Kieren G. Hollingsworth
Publicado en: Journal of Neurology, Edición 264/10, 2017, Página(s) 2053-2067, ISSN 0340-5354
Editor: Dr. Dietrich Steinkopff Verlag
DOI: 10.1007/s00415-017-8547-3

Vision DMD: Vamorolone (VBP15) drug development program for Duchenne muscular dystrophy

Autores: M. Guglieri, P. Clemens, A. Cnaan, J. Damsker, A. Arrieta, L. Morgenroth, R. Davis, C. Olsen, R. Head, K. Nagaraju, Y. Hathout, J. Haberlova, D. Athanasiou, E. Vroom, K. Bushby, E. Hoffman
Publicado en: European Journal of Paediatric Neurology, Edición 21, 2017, Página(s) e238, ISSN 1090-3798
Editor: W. B. Saunders Co., Ltd.
DOI: 10.1016/j.ejpn.2017.04.1270

New therapies in neuromuscular disorders in childhood Nové možnosti léčby vrozených neuromuskulárních onemocnění v dětském věku

Autores: MUDr. Jana Haberlová, Ph.D.
Publicado en: Neurology for Practice, Edición bimonthly, 2018, Página(s) 2018; 19(2): 108–113, ISSN 1213-1814
Editor: NEUROLOGIE PRO PRAXI

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

Autores: Eric P. Hoffman, Benjamin D. Schwartz, Laurel J. Mengle-Gaw, Edward C. Smith, Diana Castro, Jean K. Mah, Craig M. McDonald, Nancy L. Kuntz, Richard S. Finkel, Michela Guglieri, Katharine Bushby, Mar Tulinius, Yoram Nevo, Monique M. Ryan, Richard Webster, Andrea L. Smith, Lauren P. Morgenroth, Adrienne Arrieta, Maya Shimony, Catherine Siener, Mark Jaros, Phil Shale, John M. McCall, Kanneboyina Naga
Publicado en: Neurology, 2019, Página(s) 10.1212/WNL.0000000000008168, ISSN 0028-3878
Editor: Lippincott Williams & Wilkins Ltd.
DOI: 10.1212/wnl.0000000000008168

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug

Autores: Laurie S. Conklin, Jesse M. Damsker, Eric P. Hoffman, William J. Jusko, Panteleimon D. Mavroudis, Benjamin D. Schwartz, Laurel J. Mengle-Gaw, Edward C. Smith, Jean K. Mah, Michela Guglieri, Yoram Nevo, Nancy Kuntz, Craig M. McDonald, Mar Tulinius, Monique M. Ryan, Richard Webster, Diana Castro, Richard S. Finkel, Andrea L. Smith, Lauren P. Morgenroth, Adrienne Arrieta, Maya Shimony, Mark Jaros, Ph
Publicado en: Pharmacological Research, Edición 136, 2018, Página(s) 140-150, ISSN 1043-6618
Editor: Academic Press
DOI: 10.1016/j.phrs.2018.09.007

Future clinical trial design and innovative end points (Biomarkers and Imaging) for DMD and other Neuromuscular diseases workshop report 2019

Autores: Olsen, Christina; Head, Ritchie; Knerova, Jane; Larkindale, Jane; Ward, Susan; Spitali, Pietro; Kan, Hermien
Publicado en: Edición 2, 2021
Editor: Zenodo
DOI: 10.5281/zenodo.5668576

RETURNING INDIVIDUAL CLINICAL TRIAL DATA BACK TO PARTICIPANTS

Autores: Dr Laurie Conklin; Dr Holly Peay; Gaglianone, Suzanne; Suzie-Ann Bakker; Vroom, Elizabeth; Dr Eric Hoffman; Olsen, Christina
Publicado en: Edición 3, 2021
Editor: Zenodo
DOI: 10.5281/zenodo.4836583

VISION-DMD - Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy

Resultado final

Publicaciones

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